PMID- 27872728 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20200930 IS - 2035-3006 (Print) IS - 2035-3006 (Electronic) IS - 2035-3006 (Linking) VI - 8 IP - 1 DP - 2016 TI - What Unrelated Hematopoietic Stem Cell Transplantation in Thalassemia Taught us about Transplant Immunogenetics. PG - e2016048 LID - e2016048 AB - Although the past few decades have shown an improvement in the survival and complication-free survival rates in patients with beta-thalassemia major and gene therapy is already at an advanced stage of experimentation, hematopoietic stem cell transplantation (HSCT) continues to be the only effective and realistic approach to the cure of this chronic non-malignant disease. Historically, human leukocyte antigen (HLA)-matched siblings have been the preferred source of donor cells owing to superior outcomes compared with HSCT from other sources. Nowadays, the availability of an international network of voluntary stem cell donor registries and cord blood banks has significantly increased the odds of finding a suitable HLA matched donor. Stringent immunogenetic criteria for donor selection have made it possible to achieve overall survival (OS) and thalassemia-free survival (TFS) rates comparable to those of sibling transplants. However, acute and chronic graft-versus-host disease (GVHD) remains the most important complication in unrelated HSCT in thalassemia, leading to significant rates of morbidity and mortality for a chronic non-malignant disease. A careful immunogenetic assessment of donors and recipients makes it possible to individualize appropriate strategies for its prevention and management. This review provides an overview of recent insights about immunogenetic factors involved in GVHD, which seem to have a potential role in the outcome of transplantation for thalassemia. FAU - La Nasa, Giorgio AU - La Nasa G AD - Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy; Hematology Unit, Department of Medical Sciences, University of Cagliari, Italy. FAU - Vacca, Adriana AU - Vacca A AD - Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy. FAU - Littera, Roberto AU - Littera R AD - Regional Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy. FAU - Piras, Eugenia AU - Piras E AD - Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy. FAU - Orru, Sandro AU - Orru S AD - Medical Genetics, Department of Medical Sciences, University of Cagliari, Italy. FAU - Greco, Marianna AU - Greco M AD - Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy. FAU - Carcassi, Carlo AU - Carcassi C AD - Medical Genetics, Department of Medical Sciences, University of Cagliari, Italy. FAU - Caocci, Giovanni AU - Caocci G AD - Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy; Hematology Unit, Department of Medical Sciences, University of Cagliari, Italy. LA - eng PT - Journal Article PT - Review DEP - 20161020 PL - Italy TA - Mediterr J Hematol Infect Dis JT - Mediterranean journal of hematology and infectious diseases JID - 101530512 PMC - PMC5111522 COIS- Financial and Competing Interests Disclosure. The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. EDAT- 2016/11/23 06:00 MHDA- 2016/11/23 06:01 PMCR- 2016/01/01 CRDT- 2016/11/23 06:00 PHST- 2016/08/04 00:00 [received] PHST- 2016/09/16 00:00 [accepted] PHST- 2016/11/23 06:00 [entrez] PHST- 2016/11/23 06:00 [pubmed] PHST- 2016/11/23 06:01 [medline] PHST- 2016/01/01 00:00 [pmc-release] AID - mjhid-8-1-e2016048 [pii] AID - 10.4084/MJHID.2016.048 [doi] PST - epublish SO - Mediterr J Hematol Infect Dis. 2016 Oct 20;8(1):e2016048. doi: 10.4084/MJHID.2016.048. eCollection 2016.